Cerus Corporation (NASDAQ: CERS) announced today that the U.S. Food and Drug Administration (FDA) has accepted its proposed modular Premarket Approval application (PMA) shell for review of the INTERCEPT Blood System for plasma. A PMA shell is an outline of the application process that defines the structure, content and timing of each module. FDA and the applicant need to agree on a shell prior to initiation of a modular PMA submission.
”Our PMA shell proposal was designed to leverage our existing regulatory dossier from European approvals and to begin the INTERCEPT plasma regulatory submission in Q1 of 2013,” said Carol Moore, Cerus’ vice president, regulatory affairs, quality and clinical. “FDA’s agreement with our proposal means that we can target completing all four modules in 2013, putting us in a position to receive U.S. approval as early as 2014.”
A modular PMA review process is based on submission of a compilation of sections or "modules" at 90-day intervals that together become a complete PMA. Under a modular approach, FDA is able to review each module separately, allowing the applicant to receive timely feedback and potentially resolve deficiencies earlier in the review process than would be expected with a traditional PMA application.
Cerus recently announced its intention to pursue a modular PMA submission after dialogue with the FDA indicated that the company could proceed with an application for multiple indications of plasma use, not just the thrombotic thrombocytopenic purpura (TTP) indication for which Cerus received orphan drug designation last year.“Approval in the U.S. would be a pivotal achievement for us,” said Cerus' president and CEO, William ‘Obi’ Greenman. "We have already begun launch planning activities for the U.S., benefiting from our many years of experience in Europe commercializing the INTERCEPT Blood System and working with INTERCEPT plasma customers." The INTERCEPT Blood System for plasma received a CE mark in 2006, with subsequent national approvals in France, Germany and Switzerland. In clinical trials in patients with acquired coagulopathies, congenital coagulopathies and TTP, INTERCEPT plasma was well tolerated and demonstrated therapeutic efficacy comparable to conventional plasma. Data have also been collected in Phase IV studies and hemovigilance programs that support the safety and efficacy of INTERCEPT plasma in routine use across a broad variety of indications. INTERCEPT plasma is currently used by customers in Belgium, France, Germany, Italy, Kazakhstan, Russia and Spain.